BoyeS.E., BoyeS.L., PangJ.et al.2010. Functional and behavioral restoration of vision by gene therapy in the guanylate cyclase-1 (GC1) knockout mouse. PLoS One, 5:e11306.
3.
BoyeS.L., ConlonT., ErgerK.et al.2011. Long term preservation of cone photoreceptors and restoration of cone function by gene therapy in the guanylate cyclase-1 knockout (GC1KO) mouse. Invest. Ophthalmol. Vis. Sci., 52:7098–7108.
4.
MihelecM., PearsonR.A., RobbieS.J.et al.2011. Long-term preservation of Cones and Improvement in Visual function following gene therapy in a mouse model of Leber congenital amaurosis caused by guanylate cyclase-1 deficiency. Hum. Gene Ther., 22:1179–1190.
5.
MussolinoC., Della CorteM., RossiS.et al.2011. AAV-mediated photoreceptor transduction of the pig cone-enriched retina. Gene Ther., 18:637–645.
6.
TanM.H., SmithA.J., PawlykB.et al.2009. Gene therapy for retinitis pigmentosa and Leber congenital amaurosis caused by defects in AIPL1: Effective rescue of mouse models of partial and complete Aipl1 deficiency using AAV2/2 and AAV2/8 vectors. Hum. Mol. Genet., 18:2099–2114.
7.
VandenbergheL.H., BellP., MaguireA.M.et al.2011. Dosage thresholds for AAV2 and AAV8 photoreceptor gene therapy in monkey. Sci. Transl. Med., 3:88ra54.
